Abstract
Background
Several clinical trials have assessed microbiota transplant therapy (MTT) in ulcerative colitis (UC); however, none have assessed a standardized MTT product. We developed a standardized, oral, lyophilized MTT product and previously reported the primary endpoint of engraftment at week 12. Here, we assessed clinical outcomes and the association with donor engraftment.
Methods
A single-center, randomized, double-blind, placebo-controlled pilot trial of patients with UC with an endoscopic Mayo score ≥1, who received MTT or placebo for 8 weeks. Symptom and fecal assessments occurred at baseline and weeks 4, 8, and 12. Secondary outcomes included the partial Mayo score (pMS) and correlation to donor engraftment. Stool microbiota were assessed via 16S rRNA sequencing and donor engraftment measured using SourceTracker.
Results
Participants receiving MTT (n = 13) had significantly higher donor engraftment at all measured time points compared with placebo (n = 14). Compared with baseline symptoms, participants who received MTT had significantly lower pMS at weeks 4 and 8, while no significant changes in pMS were noted in those randomized to placebo. There was no correlation between donor engraftment and pMS or clinical response. There was no significant pattern in engrafting donor taxa in those that met the threshold of clinical response at week 4 vs participants that did not meet clinical response.
Conclusions
Daily dosing of encapsulated MTT for 8 weeks reduces symptoms of UC as measured by the pMS. However, donor engraftment did not correlate to pMS or clinical responses.
